Current and future therapy of hereditary angioedema due to C1 inhibitor deficiency
Complement system is classic pathway an?gen-an?body complexes ac?vate C1to form protease which cleaves C2, C4 and C3 convertase which cleaves C3 this cascade con?nue to C9, in order to stop this cascade C1 is inhibited by C1 esterase inhibitor enzyme. deficiency in this enzyme can cause angioedema.(1) Hereditary angioedema (HAE) is an autosomal dominant disease. caused by C1 esterase inhibitor deficiency. It is characterized by swelling episodes that affect the skin or mucous membranes, gastrointes?nal tract and upper airways. Laryngeal edema can be life threatening. Biochemical diagnosis can be established by diminished C4 levels and low C1 inhibitor levels or func?on. Pa?ents with HAE type I (85%) have reduced an?genic and func?onal C1 inhibitor levels, whereas pa?ents with HAE type II (15%) show only a reduc?on in C1 inhibitor func?on. During the past 2 decades, the new estrogen-dependent inherited angioedema without C1 inhibitor deficiency, also referred to as HAE type III.
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Hereditary angioedema (HAE) is an autosomal dominant disease characterized by recurrent angioedema episodes of life-threatening angioedema. A=acks of angioedema in HAE pa?ents last 3 or more days, begin during childhood, and con?nue to occur throughout life. Tragically, pa?ents with HAE con?nue to die as a direct consequence of the disease. Minimizing the morbidity and mortality associated with HAE requires both effec?ve treatment of acute a=acks as well as strategies to prevent HAE a=acks. Treatment usually involves a combina?on of prophylaxis, using androgens or an?fibroly?c drugs, and replacement with C'1 esterase inhibitor concentrate for acute a=acks and before surgery or other trauma?c procedures.This review will recap the past treatment op?ons, review the new current treatment op?ons, and discuss poten?al future treatment op?ons for pa?ents with HAE.
